Will Gene Editing Orphanize Rare Diseases?
- PMID: 36777170
- PMCID: PMC9911318
- DOI: 10.1016/j.jacbts.2022.12.002
Will Gene Editing Orphanize Rare Diseases?
References
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- Gillmore J.D., Taubel J., Gane E., et al. First-in-human in vivo CRISPR/Cas9 editing of the TTR Gene by NTLA-2001 in patients with transthyretin (ATTR) amyloidosis with cardiomyopathy. AHA 2022 Presentation Slides. American College of Cardiology. https://www.acc.org/education-and-meetings/image-and-slide-gallery/media...
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- Urnov F. We can cure disease by editing a person’s DNA. Why aren’t we? New York Times. https://www.nytimes.com/2022/12/09/opinion/crispr-gene-editing-cures.html
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- Desai N.R., Campbell C., Electricwala B., et al. Cost effectiveness of inclisiran in atherosclerotic cardiovascular patients with elevated low-density lipoprotein cholesterol despite statin use: a threshold analysis. Am J Cardiovasc Drugs. 2022;22:545–556. doi: 10.1007/s40256-022-00534-9. - DOI - PMC - PubMed
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